To make a profound impact on people’s lives, experts need to understand more about conditions and develop best-practice for care and treatments. Our commitment of over one million pounds will boost the world’s largest natural history study for Duchenne muscular dystrophy, known as NorthStar, and improve the quality of life for children and adults living with the condition. This investment will help to accelerate the development of treatments, by providing data to improve the design and evaluation of clinical trials. It also offers a unique platform to influence best practice in the care of Duchenne patients nationally.
The NorthStar study is a unique and effective consortium of all the UK neuromuscular paediatric centres. It is now expanding to also include young men and adults with Duchenne. This will help the implementation and monitoring of optimal standards of care for this particular group, which is the pre-requisite for clinical trial readiness.
Professor Francesco Muntoni, Paediatric Neurologist at Great Ormond Street Hospital:
In 2004, we helped establish NorthStar, and since then thousands of records have been added. Emerging trend-data from the study is already helping improve quality of life. By showing the benefits of steroid treatments, the NorthStar study led clinics across the country to use steroids in treating Duchenne patients. As a result, children with Duchenne are now walking for around 3.5 years longer than in the previous decade. This was identified as one of the most impressive achievements ever demonstrated at a national level.
Improvements in care over the last decade mean people with Duchenne muscular dystrophy are living longer. But these adults have less access to clinical trials than children. To address this, NorthStar will now be expanded to include the collection of information from adults, in order to develop clinical trials, and to establish guidelines for best-practice care and support.
The NorthStar network is really groundbreaking. It’s a really good resource, which shows the progress of condition and that’s very helpful for researchers. I believe it’s the international gold standard for describing boys with Duchenne, so it’s important that MDUK invests in it.
Sheila Hawkins, Muscular Dystrophy UK Trustee, who has FSHD
What is a natural history study?
A natural history study follows a group of people over time, measuring how their condition progresses and how it affects them. Healthcare professionals collect this information at every clinic appointment.
What is included in this investment?
NorthStar consists of two elements: a natural history study, and a network of consultants, physiotherapists and other healthcare professionals to support the study across the UK.
This programme focuses on a major upgrade to improve the collection of data for clinical trials. This includes investment in following roles:
- a full-time clinical co-ordinator to manage NorthStar information and work with pharmaceutical companies to share the data
- a network co-ordinator who will manage the Network
- a physiotherapist who will update and develop physiotherapy protocols and training modules
- administrative support to the 23 NorthStar Centres
- a PhD research co-ordinator who will lead on the collection of data for adults.
This programme will also see the development of the first-ever clinical care guidelines for adults with Duchenne. Improved standards of care and support could have a positive effect on life-expectancy and quality of life for men with the condition.
NorthStar has the potential to transform the lives of the 2,500 children and adults living with Duchenne in the UK.